Publications by authors named "David Cella"

785 Publications

Optimization of a technology-supported physical activity promotion intervention for breast cancer survivors: Results from Fit2Thrive.

Cancer 2021 Nov 23. Epub 2021 Nov 23.

Department of Preventive Medicine, Northwestern University Feinberg School of Medicine, Chicago, Illinois.

Background: The benefits of moderate to vigorous physical activity (MVPA) for breast cancer survivors are well established. However, most are insufficiently active. Fit2Thrive used the Multiphase Optimization Strategy methodology to determine the effect of 5 intervention components on MVPA in this population.

Methods: Two hundred sixty-nine participants (mean age, 52.5 years; SD, 9.9 years) received a core intervention (the Fit2Thrive self-monitoring app and Fitbit) and were randomly assigned to 5 intervention components set to on/off in a full factorial experiment: support calls, deluxe app, buddy, online gym, and text messages. The intervention was delivered over 12 weeks with a 12-week follow-up. MVPA was measured via accelerometry at the baseline (T1), at 12 weeks (T2), and at 24 weeks (T3). The main effects and interaction effects at each time point were examined for all components.

Results: Trial retention was high: 91.8% had valid accelerometer data at T2 or T3. Across all conditions, there were significant increases in MVPA (+53.6 min/wk; P < .001) and in the proportion of survivors meeting MVPA guidelines (+22.3%; P < .001) at T2 that were maintained but attenuated at T3 (MVPA, +24.6 min/wk; P < .001; meeting guidelines, +12.6%; P < .001). No individual components significantly improved MVPA, although increases were greater for the on level versus the off level for support calls, buddy, and text messages at T2 and T3.

Conclusions: The Fit2Thrive core intervention (the self-monitoring app and Fitbit) is promising for increasing MVPA in breast cancer survivors, but the components provided no additional increases in MVPA. Future research should evaluate the core intervention in a randomized trial and determine what components optimize MVPA behaviors in breast cancer survivors.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1002/cncr.34012DOI Listing
November 2021

Measuring Duchenne muscular dystrophy impact: development of a proxy-reported measure derived from PROMIS item banks.

Orphanet J Rare Dis 2021 Nov 22;16(1):487. Epub 2021 Nov 22.

Sarepta Therapeutics, Cambridge, MA, USA.

Background: Person-reported outcomes measurement development for rare diseases has lagged behind that of more common diseases. In studies of caregivers of patients with rare diseases, one relies on proxy report to characterize this disability. It is important to measure the child's disability accurately and comprehensively because it affects caregiver burden. We aimed to create a condition-specific caregiver proxy-report measure for Duchenne Muscular Dystrophy (DMD) in order to understand the impact of DMD on the caregiver. Drawing on relevant item banks from the Patient-Reported Outcome Measurement Information System (PROMIS), we sought to confirm their reliability and validity in the target sample of DMD caregivers.

Methods: This web-based study recruited DMD caregivers via Rare Patient Voice, patient-advocacy groups, and word of mouth. Recruitment was stratified by age of the caregiver's child with DMD, which broadly represents stages of DMD progression: 2-7, 8-12, 13-17, and > 18. Telephone interviews with DMD parent-caregivers pretested possible measures for content validity. The web-based study utilized an algorithm to categorize respondents' ambulatory status for tailored administration of PROMIS Parent-Proxy items as well as some new items developed based on caregiver interviews. Item response theory analyses were implemented.

Results: The study sample included 521 DMD caregivers representing equally the four age strata. The proxy-report measure included the following domains: fatigue impact, strength impact, cognitive function, upper extremity function, positive affect, negative affect, sleep-device symptoms, and mobility. The first five domains had strong psychometric characteristics (unidimensionality; acceptable model fit; strong standardized factor loadings; high marginal reliability). Negative Affect, covering anger, anxiety, depressive symptoms, and psychological stress, fit a bifactor model with good model fit, high marginal reliability, and strong factor loadings. The Sleep-device symptoms domain was not unidimensional, and the mobility domain did not have a simple structure due to residual correlations among items at opposite end of the mobility-disability continuum. These two domain scores were retained as clinimetric indices (i.e., uncalibrated scales), to achieve the overall goal of having a content-valid DMD-specific measure across all stages of disease severity.

Conclusions: The present study derived a DMD-specific proxy-report measure from PROMIS item banks and supplemental items that could potentially be utilized in caregiver research across all stages of the care recipient's DMD. Future research will focus on assessing the responsiveness and validity of the measure over time and its comparison to DMD patient self-report.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1186/s13023-021-02114-7DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8607700PMC
November 2021

Symptom experience of limited cutaneous systemic sclerosis from the Patients' perspective: A qualitative study.

Semin Arthritis Rheum 2021 Nov 7. Epub 2021 Nov 7.

Michigan Scleroderma Program, University of Michigan, Ann Arbor, MI, USA; Division of Rheumatology, University of Michigan, Ann Arbor, Michigan, USA.

Objectives: Limited cutaneous systemic sclerosis (lcSSc) is the largest subgroup of people with SSc, but little is known about symptom experience from the patients' perspective. This study aimed to comprehensively identify domains and symptoms reported as bothersome by patients with lcSSc and to analyze themes and sub-themes capturing symptom experience in this population.

Methods: Focus groups of patients with lcSSc were conducted using a structured guide. Patients were recruited based on an a priori purposive framework to include men and women with SSc. Focus groups were recorded, transcribed, anonymized, and analyzed using the RADaR technique (rigorous and accelerated data reduction) combined with iterative, deductive and inductive approaches.

Results: Four 2-hour Focus groups comprising participants with lcSSc were conducted (N = 26). Ninety-four symptoms were identified with 22 domains. Symptoms from the following domains were mentioned in all Focus groups: skin, musculoskeletal (MSK), cardiac, pulmonary, gastro-intestinal (GI) manifestations, fatigue, sleep, Raynaud's phenomenon (RP), pain, and digital ulcers. The three most cited domains in a pre-meeting survey were GI, RP and MSK. Seven themes defining symptom experience were identified: difficulty attributing symptoms to this complex systemic disease, influence of symptom temporality, impairment of emotional well-being, limitations in functioning (including several sub-themes of functioning), survival, symptoms management and uncertainty.

Conclusion: This study comprehensively identified domains and symptoms considered bothersome from the perspective of patients with lcSSc and summarized patient experience of lcSSc-related symptoms. This study is the first step in the design of a future combined response index dedicated to lcSSc that could foster specific research on this subpopulation of patients.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.semarthrit.2021.11.003DOI Listing
November 2021

Generation and Validation of the PROMIS Itch Questionnaire - Child to Measure the Impact of Itch on Life Quality.

J Invest Dermatol 2021 Oct 29. Epub 2021 Oct 29.

Departments of Medical Social Sciences(,) Northwestern University Feinberg School of Medicine, Chicago, IL.

Itch compromises quality of life, but most itch assessments focus only on itch intensity. We aimed to develop and validate a comprehensive PROMIS (Patient Reported Outcomes Measurement Information System®) pediatric measure for itch symptoms and itch impact, defined as the effect specifically of itch on physical, mental, and social health, all of which can affect life quality. After literature review, concept elicitation and cognitive interviews with parents and children with itch, and repeated content-expert review, an item pool was generated and refined. The pool was calibrated with data from 499 pruritic children using exploratory and confirmatory factor analyses, item response theory, and item fit analysis. The resultant 45-item bank, PROMIS Itch Questionnaire - Child (PIQ-C), showed good convergent and discriminant validity in 181 children 8-17 years of age, discriminating children with different levels of severity, and was responsive to change. Strong correlations (rho>.60) were observed with pain and sleep measures, and moderate correlations with other pediatric PROMIS measures. PIQ-C comprehensively measures itch intensity and burden, providing an itch-specific alternative for assessing life quality. The independent calibration of each item/question allows for flexibility in generating short-forms or computerized adaptive testing for efficient use in research and office practice.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.jid.2021.10.015DOI Listing
October 2021

Tolerability of bevacizumab and chemotherapy in a phase 3 clinical trial with human epidermal growth factor receptor 2-negative breast cancer: A trajectory analysis of adverse events.

Cancer 2021 Nov 2. Epub 2021 Nov 2.

Department of Social Sciences and Health Policy, Wake Forest School of Medicine, Winston-Salem, North Carolina.

Background: E5103 was a study designed to evaluate the efficacy and safety of bevacizumab. It was a negative trial for the end points of invasive disease-free survival and overall survival. The current work examines the tolerability of bevacizumab and other medication exposures with respect to clinical outcomes and patient-reported outcomes (PROs).

Methods: Adverse events (AEs) collected from the Common Terminology Criteria for Adverse Events were summarized to form an AE profile at each treatment cycle. All-grade and high-grade events were separately analyzed. The change in the AE profile over the treatment cycle was delineated as distinct AE trajectory clusters. AE-related and any-reason early treatment discontinuations were treated as clinical outcome measures. PROs were measured with the Functional Assessment of Cancer Therapy-Breast + Lymphedema. The relationships between the AE trajectory and early treatment discontinuation as well as PROs were analyzed.

Results: More than half of all AEs (57.5%) were low-grade. A cluster of patients with broad and mixed AE (all-grade) trajectory grades was significantly associated with any-reason early treatment discontinuation (odds ratio [OR], 2.87; P = .01) as well as AE-related discontinuation (OR, 4.14; P = .001). This cluster had the highest count of all-grade AEs per cycle in comparison with other clusters. Another cluster of patients with primary neuropathic AEs in their trajectories had poorer physical well-being in comparison with a trajectory of no or few AEs (P < .01). A high-grade AE trajectory did not predict discontinuations.

Conclusions: A sustained and cumulative burden of across-the-board toxicities, which were not necessarily all recognized as high-grade AEs, contributed to early treatment discontinuation. Patients with neuropathic all-grade AEs may require additional attention for preventing deterioration in their physical well-being.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1002/cncr.33992DOI Listing
November 2021

Optimizing brief, focused assessment of priority symptoms and concerns in recurrent and/or metastatic squamous cell carcinoma of the head and neck: Content validation of the Functional Assessment of Cancer Therapy/National Comprehensive Cancer Network Head and Neck Symptom Index-10 (FHNSI-10).

Health Sci Rep 2021 Dec 19;4(4):e401. Epub 2021 Oct 19.

Department of Medical Social Sciences Northwestern University Feinberg School of Medicine Chicago Illinois USA.

Background And Aims: Patients with recurrent and/or metastatic (R/M) squamous cell carcinoma of the head and neck (SCCHN) experience vast disease and treatment burdens. Brief, focused instruments are needed to assess patient-reported priority symptoms and concerns as targeted outcome assessments for use in clinical research. Although the instrument was developed based on expert and patient input and is psychometrically valid, the Functional Assessment of Cancer Therapy (FACT)/National Comprehensive Cancer Network (NCCN) Head and Neck Symptom Index-10 (FHNSI-10) has yet to undergo content validation from the perspective of R/M SCCHN patients to evaluate its use as a brief symptom-focused targeted endpoint assessment for use in clinical research.

Methods: Interviews conducted with R/M SCCHN patients explored priority symptoms and concerns, followed by cognitive debriefing of the FHNSI-10 to evaluate face validity. Transcripts were analyzed, and results were mapped to the FHNSI-10. In accordance with published recommendations, expert input from the original development and published literature was considered for content validity assessment.

Results: A total of 18 patients participated in a concept elicitation interview; saturation was obtained at interview 17. Most (83%) were undergoing active treatment, male (94%), white (72%), and did not have a college degree (67%). The most commonly mentioned symptoms were lumps/swelling, pain, sore throat, difficulty swallowing, and voice changes. For all items, ≥75% reported each question was relevant to their R/M SCCHN experience and 94% reported the instrument captured their experiences with R/M SCCHN.

Conclusion: Results provide support for the content validity of the FHNSI-10, inasmuch as all 10 items were spontaneously reported and considered relevant to R/M SCCHN. Content validity might be enhanced by adding cough and hearing impairment items; however, the existing FHNSI-10 covers the majority of symptoms uncovered in interviews with a small sample of R/M SCCHN patients.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1002/hsr2.401DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8525466PMC
December 2021

Fatigue and endocrine symptoms among women with early breast cancer randomized to endocrine versus chemoendocrine therapy: Results from the TAILORx patient-reported outcomes substudy.

Cancer 2021 Oct 6. Epub 2021 Oct 6.

Wake Forest University Health Sciences, Winston-Salem, North Carolina.

Background: TAILORx (Trial Assigning Individualized Options for Treatment) prospectively assessed fatigue and endocrine symptoms among women with early-stage hormone receptor-positive breast cancer and a midrange risk of recurrence who were randomized to endocrine therapy (E) or chemotherapy followed by endocrine therapy (CT+E).

Methods: Participants completed the Functional Assessment of Chronic Illness Therapy-Fatigue, the Patient-Reported Outcomes Measurement Information System-Fatigue Short Form, and the Functional Assessment of Cancer Therapy-Endocrine Symptoms at the baseline and at 3, 6, 12, 24, and 36 months. Linear regression was used to model outcomes on baseline symptoms, treatment, and other factors.

Results: Participants (n = 458) in both treatment arms reported greater fatigue and endocrine symptoms at early follow-up in comparison with the baseline. The magnitude of change in fatigue was significantly greater for the CT+E arm than the E arm at 3 and 6 months but not at 12, 24, or 36 months. The CT+E arm reported significantly greater changes in endocrine symptoms from the baseline to 3 months in comparison with the E arm; change scores were not significantly different at later time points. Endocrine symptom trajectories by treatment differed by menopausal status, with the effect larger and increasing for postmenopausal patients.

Conclusions: Adjuvant CT+E was associated with greater increases in fatigue and endocrine symptoms at early time points in comparison with E. These differences lessened over time, and this demonstrated early chemotherapy effects more than long-term ones. Treatment arm differences in endocrine symptoms were more evident in postmenopausal patients.

Lay Summary: Participants in TAILORx (Trial Assigning Individualized Options for Treatment) with early-stage hormone receptor-positive breast cancer and an intermediate risk of recurrence were randomly assigned to endocrine or chemoendocrine therapy. Four hundred fifty-eight women reported fatigue and endocrine symptoms at the baseline and at 3, 6, 12, 24, and 36 months. Both groups reported greater symptoms at early follow-up versus the baseline. Increases in fatigue were greater for the chemoendocrine group than the endocrine group at 3 and 6 months but not later. The chemoendocrine group reported greater changes in endocrine symptoms in comparison with the endocrine group at 3 months but not later.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1002/cncr.33939DOI Listing
October 2021

Clusters Across Multiple Domains of Health-Related Quality of Life Reveal Complex Patient Outcomes After Subarachnoid Hemorrhage.

Crit Care Explor 2021 Sep 14;3(9):e0533. Epub 2021 Sep 14.

Institute for Public Health and Medicine, Feinberg School of Medicine of Northwestern University, Chicago, IL.

Patients with aneurysmal subarachnoid hemorrhage (ruptured brain aneurysm) often have reduced health-related quality of life at follow-up in multiple domains (e.g., cognitive function and social function). We tested the hypothesis that there are distinct patterns of patient outcomes across domains of health-related quality of life, "complex patient outcomes," in survivors of subarachnoid hemorrhage.

Design: Patients with subarachnoid hemorrhage were prospectively identified. Clinical data were prospectively recorded. Health-related quality of life was prospectively assessed at 3-month follow-up using the National Institutes of Health Patient Reported Outcomes Measurement Information System and neuro-quality of life in the domains of mobility, cognitive function, satisfaction with social roles, and depression. We used k-means clustering to analyze prospectively recorded health-related quality of life data, identifying clusters of complex patient outcomes. Decision tree analysis identified index hospital stay factors predictive of a patient having a particular complex patient outcome at follow-up.

Setting: Academic medical center.

Patients: One hundred three survivors of subarachnoid hemorrhage.

Interventions: None.

Measurements And Main Results: We analyzed 103 patients, of whom 75 (72.8%) were female, and mean age was 53.6 ± 13.4 years. There were three complex patient outcomes: health-related quality of life greater than 1 sd better than the U.S. mean across all domains ( = 23, 22.3%), health-related quality of life greater than 1 sd worse than U.S. mean across all domains ( = 26, 25.2%), and satisfaction with social roles greater than 0.5 sd worse than U.S. mean with cognitive function, depression, and mobility scores near the U.S. mean ( = 54, 52.4%). In decision tree analysis, hospital disposition and Hunt and Hess Grade were associated with complex patient outcome.

Conclusions: Complex patient outcomes across multiple domains of health-related quality of life at follow-up after subarachnoid hemorrhage are distinct and may be predictable.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1097/CCE.0000000000000533DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8443826PMC
September 2021

Patient-reported outcome changes at the end of life in recurrent platinum-resistant ovarian cancer: An NRG oncology/GOG study.

Gynecol Oncol 2021 Nov 20;163(2):392-397. Epub 2021 Sep 20.

Division of Gynecologic Oncology, Summa Health System, NEOMED, Akron, OH 44310, United States of America. Electronic address:

Objectives: In a prospective study of platinum-resistant ovarian cancer patients, we examined whether the Disease-related Symptoms-Physical (DRS--P) scale of the NCCN/FACT-Ovarian Cancer Symptom Index-18 (NFOSI-18) is responsive to clinical change in patients estimated by their provider to survive at least six months.

Methods: The NFOSI-18, and other FACT measures, was collected at study entry and 3 and 6 months post-enrollment. Measures were compared for those who died or dropped off study prior to 3 months or prior to 6 months (assumed as health deterioration over time), or those who stayed on study through 6 months (presumed as stable disease over time). Statistical analyses included a fitted linear mixed model for estimating the group differences over time, Cox regression to assess the probability of survival with patient-reported outcomes, and effect size.

Results: DRS-P scores of patients who completed only one assessment were significantly lower compared to patients who were able to complete two assessments [5.9 points lower (2.0-9.8); p < 0.01], or three assessments [8.1 points lower (4.8-11.5); p < 0.01]. Measures of abdominal discomfort, functional well-being, emotional well-being, and quality of life were also significant, but treatment side effects were not. Further, in every scale except for neurotoxicity, higher (better) baseline scores were associated with a decreased likelihood of death, after adjusting for age, performance and disease status.

Conclusion: The NFOSI-18 DRS-P scale is responsive to clinical change. It has potential as an indicator of changing health status with ovarian cancer disease progression, distinct from treatment side effects.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.ygyno.2021.08.028DOI Listing
November 2021

Patient-reported outcomes predict overall survival in older patients with acute myeloid leukemia.

J Geriatr Oncol 2021 Sep 11. Epub 2021 Sep 11.

Janssen Global Services, LLC, Raritan, NJ, USA.

Introduction: Patient-reported outcomes (PROs) predict overall survival (OS) in many cancer types, but there is little evidence of their prognostic value in older patients with acute myeloid leukemia (AML). We examined whether the Functional Assessment of Cancer Therapy - Leukemia (FACT-Leu) predicted OS beyond established prognostic factors among these patients.

Materials And Methods: Data were from AML2002 (n = 309), a randomized phase 2/3 study comparing decitabine plus talacotuzumab versus decitabine alone in older AML patients ineligible for intensive chemotherapy. We used ridge-penalized Cox proportional hazards models to estimate the association between baseline FACT-Leu scales and OS. We then conducted a bootstrap analysis to determine how often FACT-Leu scales appeared in forward- and backward- selected "final models" predicting OS relative to prognosticators from the AML Composite Model (AML-CM; e.g., chronic comorbidities, previous cancer, cytogenetic/molecular risk).

Results: In ridge-penalized models, the FACT-Leu Physical Well-Being (PWB), Trial Outcomes Index (TOI), and Total scales predicted OS. Adjusting for AML-CM factors, an important increase (3 points) in PWB score was associated with a 14% reduction in the hazard of death. In the bootstrap analysis, the PWB scale appeared in 93% of backward- and 98% of forward selected models, while the TOI [57% (backward), 79% (forward)] and FACT-Leu Total [51% (backward), 78% (forward)] appeared less often in final models.

Discussion And Conclusions: These results indicate PROs' value for predicting outcomes among older AML patients and underscore the need to more systematically collect PRO data in routine care with these patients. ClinicalTrials.gov Registration: NCT02472145.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.jgo.2021.09.007DOI Listing
September 2021

Impact of Blinding on Patient-Reported Outcome Differences between Treatment Arms in Cancer Randomized Controlled Trials.

J Natl Cancer Inst 2021 Sep 11. Epub 2021 Sep 11.

University Hospital of Psychiatry II, Medical University of Innsbruck, Innsbruck, Austria.

Some concerns have been raised about potential bias in patient-reported outcome (PRO) results from open-label cancer randomized controlled trials (RCTs). We investigated if open-label trials favor the experimental treatment over the standard treatment more frequently than blinded trials. We also examined if the effect of treatment concealment differs for distal vs more proximal PROs. We assessed 538 RCTs with a PRO endpoint conducted in the most prevalent cancers, of which 366 (68.0%) were open-label, 148 (27.5%) were blinded, and 24 (4.5%) were categorized as unclear. In our multivariable logistic regression model, we did not observe a statistically significant association of the independent variable treatment concealment (open-label vs blinded) on the dependent variable measuring the proportion of trials favoring the experimental treatment (adjusted odds ratio = 1.19, 95% confidence interval = 0.79 to 1.79, 2-sided P = .40. This was also the case when comparing distal and proximal PROs. Our findings provide novel evidence-based data that support the validity of PRO results from open label cancer RCTs.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1093/jnci/djab177DOI Listing
September 2021

Turkish validation and reliability of the symptoms of Lower Urinary Tract Dysfunction Research Network Symptom Index-29 (LURN SI-29) questionnaire in patients with lower urinary tract symptoms.

Neurourol Urodyn 2021 11 9;40(8):2034-2040. Epub 2021 Sep 9.

Department of Urology, Faculty of Medicine, Istanbul Medeniyet University, Istanbul, Turkey.

Aims: To evaluate the validity and reliability of the Turkish version of the Symptoms of Lower Urinary Tract Dysfunction Research Network Symptom Index-29 (LURN SI-29).

Methods: Patients with lower urinary tract symptoms (LUTS) were included in a single-center study between January and April 2021. Patients' demographics, such as age, sex, and level of education, were recorded. The Turkish version of the LURN SI-29 and the International Prostate Symptom Score (IPSS) were administered to all patients, and the Urogenital Distress Inventory (UDI-6) was additionally administered to female patients. Construct validity was evaluated by confirmatory factor analysis. Concurrent validity was evaluated with correlations to similar measures. Internal consistency (Cronbach's alpha) and split-half reliability analyses were used to establish the scale's reliability.

Results: A total of 295 participants, 35.3% females and 64.7% males, were included in the final analysis. The mean age was 56.4 ± 11.7 years. The median total LURN SI-29 scores were 38.0 (26-50) and 26.0 (18-43) for female and male patients, respectively. The LURN SI-29 scale showed a high correlation with IPSS and UDI-6 scales (r = .758, p < .001; r = .774, p < .001, respectively) in concurrent validity analysis. Cronbach's alpha coefficient of the scale and all subscales were greater than 0.70 for both female and male patients.

Conclusions: This measurement tool can potentially be used to evaluate LUTS in Turkish women and men. Further studies should be performed to reveal the clinical usefulness of this scale.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1002/nau.24791DOI Listing
November 2021

Common patient-reported outcomes across ICHOM Standard Sets: the potential contribution of PROMIS®.

BMC Med Inform Decis Mak 2021 09 6;21(1):259. Epub 2021 Sep 6.

Amsterdam Rehabilitation Research Center | Reade, Amsterdam, The Netherlands.

Background: The International Consortium for Health Outcomes Measurement (ICHOM) develops condition-specific Standard Sets of outcomes to be measured in clinical practice for value-based healthcare evaluation. Standard Sets are developed by different working groups, which is inefficient and may lead to inconsistencies in selected PROs and PROMs. We aimed to identify common PROs across ICHOM Standard Sets and examined to what extend these PROs can be measured with a generic set of PROMs: the Patient-Reported Outcomes Measurement Information System (PROMIS®).

Methods: We extracted all PROs and recommended PROMs from 39 ICHOM Standard Sets. Similar PROs were categorized into unique PRO concepts. We examined which of these PRO concepts can be measured with PROMIS.

Results: A total of 307 PROs were identified in 39 ICHOM Standard Sets and 114 unique PROMs are recommended for measuring these PROs. The 307 PROs could be categorized into 22 unique PRO concepts. More than half (17/22) of these PRO concepts (covering about 75% of the PROs and 75% of the PROMs) can be measured with a PROMIS measure.

Conclusion: Considerable overlap was found in PROs across ICHOM Standard Sets, and large differences in terminology used and PROMs recommended, even for the same PROs. We recommend a more universal and standardized approach to the selection of PROs and PROMs. Such an approach, focusing on a set of core PROs for all patients, measured with a system like PROMIS, may provide more opportunities for patient-centered care and facilitate the uptake of Standard Sets in clinical practice.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1186/s12911-021-01624-5DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8420145PMC
September 2021

Health-related Quality of Life at the SPARTAN Final Analysis of Apalutamide for Nonmetastatic Castration-resistant Prostate Cancer Patients Receiving Androgen Deprivation Therapy.

Eur Urol Focus 2021 Aug 31. Epub 2021 Aug 31.

Massachusetts General Hospital Cancer Center and Harvard Medical School, Boston, MA, USA.

Background: In SPARTAN, apalutamide improved metastasis-free and overall survival for patients with nonmetastatic castration-resistant prostate cancer (nmCRPC) with a prostate-specific antigen doubling time of ≤10 mo.

Objective: We evaluated health-related quality of life (HRQoL) at the final analysis of the SPARTAN study.

Intervention: Patients received apalutamide (240 mg/d) or placebo in 28-d cycles. All patients continued androgen deprivation therapy (ADT).

Design, Setting, And Participants: A total of 1207 patients with nmCRPC were randomized 2:1 to apalutamide or placebo.

Outcome Measurements And Statistical Analysis: HRQoL was assessed using Functional Assessment of Cancer Therapy-Prostate (FACT-P) and EQ-5D-3L questionnaires at day 1 of cycle 1 (predose/baseline), cycles 2-6, every two cycles during cycles 7-13, every four cycles thereafter, at the end of treatment, and every 4 mo after progression to 1  yr. Results are presented using descriptive statistics. A mixed model for repeated measures was fitted to estimate the mean scores at each scheduled visit during treatment.

Results: At final analysis, with 52 mo follow-up for survival, the median treatment duration was 32.9 mo for apalutamide and 11.5 mo for placebo. Patients had good baseline HRQoL. At each scheduled collection during treatment, >90% per group completed the questionnaires. The change in FACT-P total score from baseline to cycles 21 and 25 significantly favored apalutamide over placebo (p = 0.0138 and 0.0009, respectively). The apalutamide group generally maintained favorable FACT-P (total and subscales) and EQ-5D-3L scores, while placebo scores tended to decline over time (starting in cycles 11-13 and pronounced by cycles 21-25). Notably, patient-reported fatigue did not worsen with apalutamide. Most patients reported being "not at all bothered" by side effects, and bother did not increase over time with apalutamide or placebo. Patients receiving apalutamide had minimal change in side-effect bother following symptomatic adverse events.

Conclusions: Final analysis of SPARTAN confirms that HRQoL is preserved in patients with nmCRPC receiving apalutamide plus ADT, but declines in patients receiving placebo plus ADT after approximately 1 yr.

Patient Summary: Responses from patients with prostate cancer who were included in the SPARTAN study indicated that treatment with apalutamide, even after the most extensive follow-up time possible, did not reduce their quality of life. These results, along with improved survival and longer time to the development of metastases (reported separately), confirm the benefits of apalutamide for patients with nonmetastatic castration-resistant prostate cancer.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.euf.2021.08.005DOI Listing
August 2021

User-centered development of a smartphone application (Fit2Thrive) to promote physical activity in breast cancer survivors.

Transl Behav Med 2021 Aug 16. Epub 2021 Aug 16.

Northwestern University Feinberg School of Medicine, Chicago, IL 60611, USA.

Increased moderate and vigorous physical activity (MVPA) is associated with better health outcomes in breast cancer survivors; yet, most are insufficiently active. Smartphone applications (apps) to promote MVPA have high scalability potential, but few evidence-based apps exist. The purpose is to describe the testing and usability of Fit2Thrive, a MVPA promotion app for breast cancer survivors. A user-centered, iterative design process was utilized on three independent groups of participants. Two groups of breast cancer survivors (group 1 n = 8; group 2: n = 14) performed app usability field testing by interacting with the app for ≥3 days in a free-living environment. App refinements occurred following each field test. The Post-Study System Usability Questionnaire (PSSUQ) and the User Version Mobile Application Rating Scale (uMARS) assessed app usability and quality on a 7- and 5-point scale, respectively, and women provided qualitative written feedback. A third group (n = 15) rated potential app notification content. Quantitative data were analyzed using descriptive statistics. Qualitative data were analyzed using a directed content analysis. The PSSUQ app usability score (M1= 3.8; SD = 1.4 vs. M2= 3.2; SD = 1.1; lower scores are better) and uMARS app quality score (M1 = 3.4; SD = 1.3 vs. M2= 3.4; SD = 0.6; higher scores are better) appeared to improve in Field Test 2. Group 1 participants identified app "clunkiness," whereas group 2 participants identified issues with error messaging/functionality. Group 3 "liked" 53% of the self-monitoring, 71% of the entry reminder, 60% of the motivational, and 70% of the goal accomplishment notifications. Breast cancer survivors indicated that the Fit2Thrive app was acceptable and participants were able to use the app. Future work will test the efficacy of this app to increase MVPA.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1093/tbm/ibab112DOI Listing
August 2021

Comparison of PROMIS Computerized Adaptive Testing versus Fixed Short Forms in Juvenile Myositis.

Arthritis Care Res (Hoboken) 2021 Jul 30. Epub 2021 Jul 30.

Division of Pediatric Rheumatology, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.

Objective: Patient-Reported Outcomes Measurement Information System (PROMIS ) measures can be administered via computerized adaptive testing (CAT) or fixed short forms (FSF), but the empirical benefits of CAT versus FSF are unknown in juvenile myositis (JM). This study assesses if PROMIS CAT is feasible, precise, correlated with FSF, and less prone to respondent burden and floor/ceiling effects than FSF in JM.

Methods: Patients 8-17 yo (self-report and parent proxy) and parents of patients 5-7 yo (only parent proxy) completed PROMIS Fatigue, Pain Interference, Upper Extremity Function, Mobility, Anxiety and Depressive Symptoms measures. Pearson correlations, paired t-tests, and Cohen's D were calculated between PROMIS CAT and FSF. McNemar test assessed floor/ceiling effects between CAT and FSF. Precision and respondent burden were examined across the T-score range.

Results: Data from 67 patient-parent dyads were analyzed. CAT and FSF mean scores did not significantly differ except in parent proxy Anxiety and Fatigue (effect size: 0.23 and 0.19, respectively). CAT had less pronounced floor/ceiling effects at the less symptomatic extreme in all domains except self-report Anxiety. Increased item burden and higher standard errors were seen in less symptomatic scorers for CAT. Modified stopping rules limiting CAT item administration did not decrease precision.

Conclusion: PROMIS CAT appears to be feasible and correlated with FSF. CAT had less pronounced floor/ceiling effects, allowing detection of individual differences in less symptomatic patients. Modified stopping rules for CAT may decrease respondent burden. CAT can be considered for long-term follow-up of JM patients.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1002/acr.24760DOI Listing
July 2021

Considerations for a combined index for limited cutaneous systemic sclerosis to support drug development and improve outcomes.

J Scleroderma Relat Disord 2021 Feb 5;6(1):66-76. Epub 2020 Oct 5.

Department of Internal Medicine, Division of Rheumatology, Scleroderma Program, University of Michigan, Ann Arbor, Michigan, USA.

Systemic sclerosis (SSc; systemic scleroderma) is characterized by a heterogeneous range of clinical manifestations. SSc is classified into limited cutaneous SSc (lcSSc) and diffuse cutaneous subgroups (dcSSc) based on the extent of skin involvement. Randomized controlled trials in scleroderma have mainly focused on dcSSc partly because the measurement of skin involvement, critical for evaluating a therapeutic intervention is more dynamic in this subset. Nonetheless, lcSSc, the most common cutaneous subset (about 2/3), is also associated with significant morbidity and detrimental impact on health-related quality of life. The lack of interventional studies in lcSSc is partly due to a lack of relevant outcome measures to evaluate this subgroup. Combining several clinically meaningful outcomes selected specifically for lcSSc may improve representativeness in clinical trials and responsiveness of outcomes measured in randomized controlled trials. A composite index dedicated to lcSSc combining such relevant outcomes could advance clinical trial development for lcSSc by providing the opportunity to test and select among candidate drugs that could act as disease-modifying treatments for this neglected subgroup of SSc. This proposed index would include items selected by expert physicians and patients with lcSSc across domains grounded in the lived experience of lcSSc. This article reviews the reasons behind the relative neglect of lcSSc, discusses the current state of outcome measures for lcSSc, identifies challenges, and proposes a roadmap for a combined lcSSc-specific treatment response index.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1177/2397198320961967DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8313014PMC
February 2021

Meaning and purpose in Huntington's disease: a longitudinal study of its impact on quality of life.

Ann Clin Transl Neurol 2021 08 20;8(8):1668-1679. Epub 2021 Jul 20.

Department of Physical Medicine and Rehabilitation, University of Michigan, Ann Arbor, Michigan, USA.

Objective: Previous work in Huntington's disease (HD) has shown that a sense of meaning and purpose (M&P) is positively associated with positive affect and well-being (PAW); however, it was unknown whether HD-validated patient-reported outcomes (PROs) influence this association and how M&P impacts PROs in the future. Our study was designed to examine if HD-validated PROs moderate the relationship between M&P and PAW and to evaluate if baseline M&P predicts 12- and 24-month changes in HD-validated PROs.

Methods: This was a longitudinal, multicenter study to develop several PROs (e.g., specific for the physical, emotional, cognitive, and social domains) for people with HD (HDQLIFE). The sample consisted of 322 people with HD (n = 50 prodromal, n = 171 early-stage manifest, and n = 101 late-stage manifest HD). A single, multivariate linear mixed-effects model was performed with PAW as the outcome predicted by main effects for M&P and several moderators (i.e., an HD-validated PRO) and interactions between M&P and a given PRO. Linear-mixed models were also used to assess if baseline M&P predicted HD-validated PROs at 12 and 24 months.

Results: Higher M&P was positively associated with higher PAW regardless of the magnitude of symptom burden, as represented by HD-validated PROs, and independent of disease stage. In our primary analysis, baseline M&P predicted increased PAW and decreased depression, anxiety, anger, emotional/behavioral disruptions, and cognitive decline at 12 and 24 months across all disease stages.

Interpretation: These findings parallel those seen in the oncology population and have implications for adapting and developing psychotherapeutic and palliative HD interventions.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1002/acn3.51424DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8351386PMC
August 2021

Reliability, validity and important difference estimates for the NCCN-FACT Ovarian Symptom Index-18 (NFOSI-18).

Future Oncol 2021 Oct 21;17(30):3951-3964. Epub 2021 Jul 21.

Adelphi Values, Bollington, UK.

To evaluate psychometric performance of the NCCN-FACT Ovarian Cancer Symptom Index-18 (NFOSI-18) in advanced ovarian cancer. Cross-sectional, observational data from patients receiving treatment for ovarian cancer. Other measures included European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire core (EORTC QLQ-C30) and associated ovarian cancer module (EORTC QLQ-OV28) and Work Productivity and Activity Impairment. Internal consistency reliability, construct validity and anchor-based clinically important differences were assessed. 897 patients were analyzed. Reliability was acceptable for all NFOSI-18 scores; construct validity was supported. Twelve anchors sufficiently correlated with NFOSI-18 scores and suggested clinically important differences: NFOSI-18 total score (5-7), disease-related symptoms - physical (3-4), disease-related symptoms - emotional (1), treatment side effects (2) and functional well-being (1-2). Results provide evidence of reliability and validity of NFOSI-18 scores. Generated CIDs will help improve interpretation of between-group treatment differences in clinical trials.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.2217/fon-2021-0358DOI Listing
October 2021

Correlates of financial toxicity in adult cancer patients and their informal caregivers.

Support Care Cancer 2021 Jul 13. Epub 2021 Jul 13.

Department of Radiology, University of Michigan, Ann Arbor, MI, USA.

Background: Financial toxicity is commonly reported by cancer patients, but few studies have assessed caregiver perceptions. We aimed to validate the modified Comprehensive Score for Financial Toxicity (COST) in cancer caregivers, identify factors associated with financial toxicity in both patients and caregivers, and assess the association of caregiver financial toxicity with patient and caregiver outcomes.

Methods: Using a convenience sampling method, 100 dyads of adult cancer patients and a primary caregiver visiting outpatient oncology clinics (Jan-Sep 2019) were recruited. We assessed the internal consistency and convergent and divergent validity of the modified COST. Multivariable analyses identified correlates of financial toxicity. Association of financial toxicity with care non-adherence, lifestyle-altering behaviors (e.g., home refinance/sale, retirement/saving account withdrawal), and quality of life (QOL) was investigated.

Results: Recruited patient vs. caregiver characteristics were as follows: mean age: 60.6 vs. 56.5; 34% vs. 46.4% female; 79% vs. 81.4% white. The caregiver COST measure demonstrated high internal consistency (Cronbach α = 0.91). In patients, older age (B, 0.3 [95% CI, 0.1-0.4]) and higher annual household income (B, 14.3 [95% CI, 9.3-19.4]) correlated with lower financial toxicity (P < 0.05). In caregivers, lower patient financial toxicity (B, 0.4 [95% CI, 0.2-0.6]) and cancer stages 1-3 (compared to stage 4) (B, 4.6 [95% CI, 0.4-8.8]) correlated with lower financial toxicity (P < 0.05). Increased caregiver financial toxicity correlated with higher care non-adherence in patients, increased lifestyle-altering behaviors, and lower QOL in patients and caregivers (P < 0.05).

Conclusion: The COST measure can also be used to assess caregiver financial toxicity. Caregivers' financial toxicity was associated with negative outcomes for both dyad members.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1007/s00520-021-06424-1DOI Listing
July 2021

Minimal important change (MIC): a conceptual clarification and systematic review of MIC estimates of PROMIS measures.

Qual Life Res 2021 Oct 10;30(10):2729-2754. Epub 2021 Jul 10.

Department of Epidemiology and Data Science, Amsterdam Public Health Research Institute, Amsterdam UMC, Vrije Universiteit Amsterdam, P.O. box 7057, 1007, Amsterdam, MB, The Netherlands.

We define the minimal important change (MIC) as a threshold for a minimal within-person change over time above which patients perceive themselves importantly changed. There is a lot of confusion about the concept of MIC, particularly about the concepts of minimal important change and minimal detectable change, which questions the validity of published MIC values. The aims of this study were: (1) to clarify the concept of MIC and how to use it; (2) to provide practical guidance for estimating methodologically sound MIC values; and (3) to improve the applicability of PROMIS by summarizing the available evidence on plausible PROMIS MIC values. We discuss the concept of MIC and how to use it and provide practical guidance for estimating MIC values. In addition, we performed a systematic review in PubMed on MIC values of any PROMIS measure from studies using recommended approaches. A total of 50 studies estimated the MIC of a PROMIS measure, of which 19 studies used less appropriate methods. MIC values of the remaining 31 studies ranged from 0.1 to 12.7 T-score points. We recommend to use the predictive modeling method, possibly supplemented with the vignette-based method, in future MIC studies. We consider a MIC value of 2-6 T-score points for PROMIS measures reasonable to assume at this point. For surgical interventions a higher MIC value might be appropriate. We recommend more high-quality studies estimating MIC values for PROMIS.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1007/s11136-021-02925-yDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8481206PMC
October 2021

Cognitive dysfunction prevalence and associated factors in older breast cancer survivors.

J Geriatr Oncol 2021 Jul 8. Epub 2021 Jul 8.

Indiana University School of Nursing, 600 Barnhill Drive, Indianapolis, IN 46202, USA. Electronic address:

Objectives: The purpose of this study was to examine the prevalence and factors associated with objective and subjective cognitive dysfunction in older breast cancer survivors (BCS).

Materials And Methods: This cross-sectional descriptive study leveraged previously collected data from older BCS (n = 335). Separate linear regression models were used to determine relationships between demographic factors (age, education), medical factors (comorbidities), disease factors (time since diagnosis, cancer stage), cancer-related symptoms (depressive symptoms, anxiety, fatigue, sleep disturbance) and cognitive dysfunction measures, including objective learning, delayed recall, attention, executive function-working memory, verbal fluency and subjective attentional function.

Results: Cognitive dysfunction was prevalent with up to 18.6% of older BCS experiencing mild-moderate dysfunction (1.5 standard deviations below mean of non-cancer controls) in at least one cognitive domain. Poor to moderate subjective attentional function was reported by 26% of older BCS. More depressive symptoms were significantly related to poorer cognitive function including learning (p < .01), delayed recall (p < .05), verbal fluency (p < .001), and subjective attentional function (p < .001) but not attention and executive function-working memory. Age, education, anxiety, and fatigue were also negatively associated with cognitive function in some models (p < .05-0.001).

Conclusion: Cognitive dysfunction is common among older BCS and depressive symptoms, anxiety, and fatigue are related factors. Importantly, depressive symptoms were not only related to self-report, but also to cognitive performance. Healthcare providers should be aware of and assess for related factors and cognitive dysfunction itself in older BCS even years after diagnosis and treatment thorough geriatric assessment. Future longitudinal research is needed to discern these relationships.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.jgo.2021.07.001DOI Listing
July 2021

Patient-Reported Outcomes in the Phase III BRIGHTE Trial of the HIV-1 Attachment Inhibitor Prodrug Fostemsavir in Heavily Treatment-Experienced Individuals.

Patient 2021 Jun 28. Epub 2021 Jun 28.

ViiV Healthcare, Branford, CT, USA.

Introduction: Heavily treatment-experienced (HTE) people living with HIV-1 (PLWH) have limited viable antiretroviral regimens available because of multidrug resistance and safety concerns. The first-in-class HIV-1 attachment inhibitor fostemsavir demonstrated efficacy and safety in HTE participants in the ongoing phase III BRIGHTE trial.

Objectives: We describe patient-reported outcomes (PROs) through week 48.

Methods: Eligible participants for whom their current regimen was failing were assigned to the randomized cohort (RC; one to two fully active agents remaining) or the nonrandomized cohort (NRC; no fully active agents remaining). PRO assessments included the EQ-5D-3L, EQ-VAS, and Functional Assessment of HIV Infection (FAHI) instruments.

Results: Both cohorts achieved increases in EQ-5D-3L US- and UK-referenced utility score from baseline at week 24. Mean visual analog scale (VAS) scores in the RC and NRC increased from baseline by 8.7 (95% CI 6.2-11.2) and 5.6 points (95% CI 1.5-9.7) at week 24 and increased from baseline by 9.8 (95% CI 7.0-12.6) and 4.9 points (95% CI 0.6-9.2) at week 48, respectively. Mean increases in FAHI total score from baseline to weeks 24 and 48 in the RC were 6.9 (95% CI 4.2-9.7) and 5.8 (95% CI 2.7-9.0), respectively, whereas mean increases in physical and emotional well-being subscale scores were 2.7 (95% CI 1.9-3.6) and 2.4 (95% CI 1.3-3.4) and 3.2 (95% CI 2.2-4.2) and 2.6 (95% CI 1.6-3.7), respectively, with little to no change in other subscales.

Conclusions: Improvements in major domains of the EQ-VAS and FAHI through week 48, combined with efficacy and safety results, support the use of fostemsavir for HTE PLWH.

Trial Registration Number And Date: NCT02362503; February 13, 2015.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1007/s40271-021-00534-yDOI Listing
June 2021

Association of cognitive impairment and breast cancer survivorship on quality of life in younger breast cancer survivors.

J Cancer Surviv 2021 Jun 26. Epub 2021 Jun 26.

IU Simon Cancer Center, Indiana University School of Nursing, 600 Barnhill Drive, NU 318, Indianapolis, IN, 46202, USA.

Purpose: Younger breast cancer survivors (BCS) often report cognitive impairment and poor quality of life (QoL), which could be interrelated. The purpose of this study was to examine the association of cognitive impairment and breast cancer status (BCS versus healthy control (HC)), with QoL, which included psychological (depressive symptoms, well-being, perceived stress, and personal growth) and physical well-being (physical functioning and fatigue).

Methods: Four hundred ninety-eight BCS (≤45 years at diagnosis) who were 3 to 8 years post-chemotherapy treatment and 394 HC completed subjective questionnaires and a one-time neuropsychological assessment, including tests of attention, memory, processing speed, and verbal fluency. For each test, cognitive impairment was defined as scoring 1.5 and 2.0 standard deviations below the mean of the HC group. Separate linear regression models for each outcome were ran controlling for known covariates.

Results: BCS reported significantly more memory problems than HC (p < 0.0001), with up to 23% having significant impairment. Cognitive performance did not differ significantly between BCS and HCs. BCS vs. HCs had greater depression and fatigue, yet more personal growth. Objective and subjective cognitive impairment were significantly related to greater depressive symptoms and perceived stress and lower well-being and physical functioning; whereas, objective impairment was related to less personal growth and subjective impairment was related to greater fatigue.

Conclusions: Younger BCS report significant cognitive impairment years after treatment which may relate to greater decrements in QoL.

Implications To Cancer Survivors: Assessment and interventions to address cognitive concerns may also influence QoL outcomes in younger BCS.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1007/s11764-021-01075-xDOI Listing
June 2021

Linking Scores with Patient-Reported Health Outcome Instruments:A VALIDATION STUDY AND COMPARISON OF THREE LINKING METHODS.

Psychometrika 2021 09 26;86(3):717-746. Epub 2021 Jun 26.

Department of Educational Psychology, The University of Texas at Austin, 1912 Speedway, Stop D5800, Austin, TX, 78712-1289, USA.

The psychometric process used to establish a relationship between the scores of two (or more) instruments is generically referred to as linking. When two instruments with the same content and statistical test specifications are linked, these instruments are said to be equated. Linking and equating procedures have long been used for practical benefit in educational testing. In recent years, health outcome researchers have increasingly applied linking techniques to patient-reported outcome (PRO) data. However, these applications have some noteworthy purposes and associated methodological questions. Purposes for linking health outcomes include the harmonization of data across studies or settings (enabling increased power in hypothesis testing), the aggregation of summed score data by means of score crosswalk tables, and score conversion in clinical settings where new instruments are introduced, but an interpretable connection to historical data is needed. When two PRO instruments are linked, assumptions for equating are typically not met and the extent to which those assumptions are violated becomes a decision point around how (and whether) to proceed with linking. We demonstrate multiple linking procedures-equipercentile, unidimensional IRT calibration, and calibrated projection-with the Patient-Reported Outcomes Measurement Information System Depression bank and the Patient Health Questionnaire-9. We validate this link across two samples and simulate different instrument correlation levels to provide guidance around which linking method is preferred. Finally, we discuss some remaining issues and directions for psychometric research in linking PRO instruments.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1007/s11336-021-09776-zDOI Listing
September 2021

Strategies for Effective Implementation of Patient-Reported Outcome Measures in Arthroplasty Practice.

J Bone Joint Surg Am 2021 Jun 18. Epub 2021 Jun 18.

Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, Illinois.

Abstract: Patient-reported outcome measures (PROMs) will be an important component of real-world evidence, but best practices for capture and integration are not yet defined. While digital tools support patients, clinicians, and researchers to collect PROMs, PROM capture in clinical care remains challenging. We synthesized PROM implementation strategies that are successfully used by hundreds of arthroplasty surgeons and early PROM-adopter clinical systems. This information can guide health systems that are preparing to implement PROMs to inform clinical care, drive quality-improvement activities, and support reporting for payer-sponsored incentives. Specific information is included to guide each step in the implementation process, including selecting PROMs, redesigning office procedures, programming of information technology, and informing interpretation and clinical use. While no one solution exists for successful PROM implementation in total hip and total knee replacement, referred to as total joint replacement, these guidelines can inform optimal PROM deployment and the capture of complete PROM data. In addition, we outline future research that is needed to define methods for optimal patient engagement, technology infrastructure, and operational systems to seamlessly integrate PROM collection in clinical care.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.2106/JBJS.20.02072DOI Listing
June 2021

Association of Modifiable Risk Factors With Early Discontinuation of Adjuvant Endocrine Therapy: A Post Hoc Analysis of a Randomized Clinical Trial.

JAMA Oncol 2021 Jun 17. Epub 2021 Jun 17.

Department of Social Sciences and Health Policy, Wake Forest University Health Sciences, Winston Salem, North Carolina.

Importance: Early discontinuation of adjuvant endocrine therapy (ET) is problematic among breast cancer survivors, with previous studies suggesting that up to 50% of women do not adhere to the recommended full 5 years of ET treatment.

Objective: To identify the association between early discontinuation of ET in the Trial Assigning Individualized Options for Treatment (TAILORx) and modifiable risk factors, polypharmacy, and types of additional medications such as antidepressants and opioids.

Design, Setting, And Participants: This post hoc analysis includes a subgroup of 954 patients with breast cancer in TAILORx, a randomized clinical trial conducted from April 7, 2006, to October 6, 2010. All participants received a diagnosis of hormone receptor-positive, ERBB2-negative, axillary node-negative breast cancer and started ET within a year of study entry. Analyses were conducted in the intent-to-treat population. Statistical analysis took place from January 15, 2020, to April 6, 2021.

Main Outcomes And Measures: Participants completed measures on cancer-related health-related quality of life including physical well-being and social well-being prior to initiating ET. Early discontinuation of ET was defined as discontinuation less than 4 years from initiation for reasons other than death or recurrence. Kaplan-Meier estimates were used to calculate discontinuation, and Cox proportional hazards regression joint prediction models were used to analyze the association between rates of adherence to ET with patient-level factors.

Results: A total of 954 women (mean [SD] age, 56.6 [8.9] years) were included in this analysis. In a joint model, receipt of chemoendocrine therapy (vs receipt of ET only; hazard ratio [HR], 0.57; 95% CI, 0.35-0.92; P = .02) and age older than 40 years (vs ≤40 years; HR for 41-50 years, 0.39; 95% CI, 0.18-0.85; P = .02; HR for 51-60 years, 0.28; 95% CI, 0.13-0.60; P = .001; HR for 61-70 years, 0.40; 95% CI, 0.18-0.86; P = .02; and HR for >70 years, 0.23; 95% CI, 0.07-0.77; P = .02) were associated with a lower probability of early discontinuation of ET. Adjusted for these factors, a history of depression compared with no history of depression (HR, 1.82; 95% CI, 1.19-2.77; P = .005), worse physical well-being compared with better physical well-being (HR, 2.12; 95% CI, 1.30-3.45; P = .002), and worse social well-being compared with better social well-being (HR, 1.94; 95% CI, 1.20-3.13; P = .006) were individually and significantly associated with a higher probability of early discontinuation of ET. Only antidepressant use at study baseline was associated with early discontinuation (HR, 1.87; 95% CI, 1.23-2.84; P = .003).

Conclusions And Relevance: In this post hoc analysis of a randomized clinical trial, baseline patient-reported health-related quality of life components, such as poor social well-being, poor physical well-being, and comorbid depression, were significant risk factors for early discontinuation of endocrine therapies. These results support systematic screening for patient-reported outcomes and depressive symptoms to identify women at risk for discontinuation of ET.

Trial Registration: ClinicalTrials.gov Identifier: NCT00310180.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1001/jamaoncol.2021.1693DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8377561PMC
June 2021

The Functional Assessment of Cancer Therapy Eight Dimension (FACT-8D), a Multi-Attribute Utility Instrument Derived From the Cancer-Specific FACT-General (FACT-G) Quality of Life Questionnaire: Development and Australian Value Set.

Value Health 2021 Jun 7;24(6):862-873. Epub 2021 May 7.

Northwestern University Feinberg School of Medicine, Department of Medical Social Sciences, Chicago, IL, USA.

Objectives: To develop a cancer-specific multi-attribute utility instrument derived from the Functional Assessment of Cancer Therapy - General (FACT-G) health-related quality of life (HRQL) questionnaire.

Methods: We derived a descriptive system based on a subset of the 27-item FACT-G. Item selection was informed by psychometric analyses of existing FACT-G data (n = 6912) and by patient input (n = 82). We then conducted an online valuation survey, with participants recruited via an Australian general population online panel. A discrete choice experiment (DCE) was used, with attributes being the HRQL dimensions of the descriptive system and survival duration, and 16 choice-pairs per participant. Utility decrements were estimated with conditional logit and mixed logit modeling.

Results: Eight HRQL dimensions were included in the descriptive system: pain, fatigue, nausea, sleep, work, social support, sadness, and future health worry; each with 5 levels. Of 1737 panel members who accessed the valuation survey, 1644 (95%) completed 1 or more DCE choice-pairs and were included in analyses. Utility decrements were generally monotonic; within each dimension, poorer HRQL levels generally had larger utility decrements. The largest utility decrements were for the highest levels of pain (-0.40) and nausea (-0.28). The worst health state had a utility of -0.54, considerably worse than dead.

Conclusions: A descriptive system and preference-based scoring approach were developed for the FACT-8D, a new cancer-specific multi-attribute utility instrument derived from the FACT-G. The Australian value set is the first of a series of country-specific value sets planned that can facilitate cost-utility analyses based on items from the FACT-G and related FACIT questionnaires containing FACT-G items.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.jval.2021.01.007DOI Listing
June 2021

Can the Knee Outcome and Osteoarthritis Score (KOOS) Function Subscale Be Linked to the PROMIS Physical Function to Crosswalk Equivalent Scores?

Clin Orthop Relat Res 2021 12;479(12):2653-2664

Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.

Background: An increased focus on patient-reported outcome measures (PROMs) has led to a proliferation of these measures in orthopaedic surgery. Mandating a single PROM in clinical and research orthopaedics is not feasible given the breadth of data already collected with older measures and the emergence of psychometrically superior measures. Creating crosswalk tables for scores between measures allows providers to maintain control of measure choice. Furthermore, crosswalk tables permit providers to compare scores collected with older outcome measures with newly collected ones. Given the widespread use of the newer Patient-reported Outcome Measure Information System Physical Function (PROMIS PF) and the established Knee Outcome and Osteoarthritis Score (KOOS), it would be clinically useful to link these two measures.

Question/purpose: Can the KOOS Function in Activities of Daily Living (ADL) subscale be robustly linked to the PROMIS PF to create a crosswalk table of equivalent scores that accurately reflects a patient's reported physical function level on both scales?

Methods: We sought to establish a common standardized metric for collected responses to the PROMIS PF and the KOOS ADL to develop equations for converting a PROMIS PF score to a score for the KOOS-ADL subscale and vice versa. To do this, we performed a retrospective, observational study at two academic medical centers and two community hospitals in an urban and suburban healthcare system. Patients 18 years and older who underwent TKA were identified. Between January 2017 and July 2020, we treated 8165 patients with a TKA, 93% of whom had a diagnosis of primary osteoarthritis. Of those, we considered patients who had completed a full KOOS and PROMIS PF 10a on the same date as potentially eligible. Twenty-one percent (1708 of 8165) of patients were excluded because no PROMs were collected at any point, and another 67% (5454 of 8165) were excluded because they completed only one of the required PROMs, leaving 12% (1003 of 8165) for analysis here. PROMs were collected each time they visited the health system before and after their TKAs. Physical function was measured by the PROMIS PF version 1.0 SF 10a and KOOS ADL scale. Analyses to accurately create a crosswalk of equivalent scores between the measures were performed using the equipercentile linking method with both unsmoothed and log linear smoothed score distributions.

Results: Crosswalks were created, and adequate validation results supported their validity; we also created tables to allow clinicians and clinician scientists to convert individual patients' scores easily. The mean difference between the observed PROMIS PF scores and the scores converted by the crosswalk from the KOOS-ADL scores was -0.08 ± 4.82. A sensitivity analysis was conducted, confirming the effectiveness of these crosswalks to link the scores of two measures from patients both before and after surgery.

Conclusion: The PROMIS PF 10a can be robustly linked to the KOOS ADL measure. The developed crosswalk table can be used to convert PROMIS PF scores from KOOS ADL and vice versa.

Clinical Relevance: The creation of a crosswalk table between the KOOS Function in ADL subscale and PROMIS PF allows clinicians and researchers to easily convert scores between the measures, thus permitting greater choice in PROM selection while preserving comparability between patient cohorts and PROM data collected from older outcome measures. Creating a crosswalk, or concordance table, between the two scales will facilitate this comparison, especially when pooling data for meta-analyses.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1097/CORR.0000000000001857DOI Listing
December 2021

Developing a common metric for depression across adulthood: Linking PROMIS depression with the Edinburgh Postnatal Depression Scale.

Psychol Assess 2021 Jul 31;33(7):610-618. Epub 2021 May 31.

Department of Medical Social Sciences.

Depression is a leading mental health concern across the U.S. and worldwide. There are myriad assessments to evaluate depressive symptoms, including the Edinburgh Postnatal Depression Scale (EPDS), which is widely used to evaluate women's pre- and postnatal depression but not as prevalent at other timepoints in adulthood, limiting its utility for longitudinal research. As part of the National Institutes of Health's (NIH) Environmental influences on Child Health Outcomes (ECHO) Research Program, the current study sought to develop a common metric so that scores on the EPDS can be converted to the standardized Patient-Reported Measurement Information System (PROMIS®) T-score metric. Drawing on data from the ECHO-Prenatal Alcohol in SIDS and Stillbirth cohort, this study used a single-group linking design, where 1,263 mothers completed the EPDS and PROMIS-Depression measures at the same time. Score linking was conducted using equipercentile and item response theory (IRT) methods. Results showed both linking methods provide robust, congruent results, and subgroup invariance held across age, race, ethnicity, education, and geographic location. The IRT-based unidimensional fixed-parameter calibration was selected due to its model simplicity, and a crosswalk table was established to convert scores from the EPDS to PROMIS T-scores. Overall, this study provides a way to aggregate data across various depression measures and timepoints, such that researchers and clinicians now have the ability to directly compare and combine EPDS data with PROMIS and other depression measures already score-linked to PROMIS. (PsycInfo Database Record (c) 2021 APA, all rights reserved).
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1037/pas0001009DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8284177PMC
July 2021
-->